J&J to Host Investor Conference Call on the Pharmaceutical Business

Johnson & Johnson (NYSE: JNJ) will host a conference call for investors at 8:00 a.m. (Eastern Time) on Thursday, September 13^th, to review its Pharmaceutical Business.   Joaquin Duato, Vice Chairman of the Executive Committee; Jennifer Taubert, Executive Vice President, Worldwide Chairman, Pharmaceuticals; Mathai Mammen, Global Head, Janssen Research & Development and Lesley Fishman, Senior Director Investor Relations will host the call.

Investors and other interested parties can access the webcast/conference call in the following ways:

• The webcast and presentation material are accessible at Johnson & Johnson’s website www.investor.jnj.com. A replay of the webcast will be available approximately three hours after the conference call concludes.
• By telephone: for both “listen-only” participants and those financial analysts who wish to take part in the question-and-answer portion of the call, the telephone dial-in number in the U.S. is 877-869-3847. For participants outside the U.S., the dial-in number is 201-689-8261.
• A replay of the conference call will be available until approximately 12:00 a.m. on September 21, 2018. The replay dial-in number for U.S. participants is 877-660-6853. For participants outside the U.S., the replay dial-in number is 201-612-7415. The replay conference ID number for all callers is 13681049.

https://bit.ly/2L9cBwu

Poised for phase 3, Galera med wards off radiation side effects

For many head and neck cancer patients who receive radiation therapy, side effects, such as oral mucositis, are a fact of life. This inflammatory condition cannot be prevented and can only be treated once it develops, but a synthetic enzyme from Galera Therapeutics could change that.

In data to be presented Monday at a meeting of the American Chemical Society, the enzyme mimetic, dubbed GC4419, appears to stave off severe oral mucositis (SOM), a condition in which the epithelial cells lining the gastrointestinal tract break down. SOM opens the mucus membranes to ulceration and infection, which in turn can lead to further side effects. Because they cannot eat, patients with SOM may need feeding tubes as well as narcotic analgesics to ease their pain. The condition can also be dose-limiting; that is, subsequent chemotherapy may have to be given in reduced doses or delayed altogether.

Radiation triggers a rise in superoxide radical levels, which are thought to lead to oral mucositis and other side effects. In the study, the researchers showed that GC4419 converts superoxide to molecular oxygen and hydrogen peroxide. The enzyme could deliver a one-two punch against cancer—in addition to getting rid of superoxide, it increases levels of hydrogen peroxide, which kills cancer cells.

“The synthetic enzyme we designed and made mimics the function of the naturally occurring superoxide dismutase, an enzyme that converts superoxide to molecular oxygen and hydrogen peroxide,” Riley said. “Hydrogen peroxide is very toxic to cancer cells but not to normal cells. Thus, we create two opportunities to improve radiation therapy: reducing toxicity for normal cells while increasing the toxicity to the cancerous ones.”

GC4419 is just one of several dismutase mimetics in Galera’s pipeline.

“We had to figure out which isomer would work best and be the most ‘druggable,'” Riley said. “Our current synthesis involves using a template to make the 15-membered macrocylic ring complex in a single GMP [Good Manufacturing Practice] step. It uses pyridine-2,6- dicarboxyaldehyde, manganese(II) chloride and a linear tetramine synthesized from S,S-1,2 diaminocyclohexane. It is an elegantly simple ‘one-pot’ synthesis that yields the desired molecule with more than 99.5 percent chemical purity.”

GC4419 has completed phase 1 and 2 trials and is poised to enter phase 3 this year. If approved, it could be used to ward off SOM and its side effects not just in patients with head and neck cancers, but in those with other types of cancer.

For example, it may be useful in pancreatic cancer, which is often diagnosed at late stages and is hard to treat because of the pancreas’ location deep within the abdomen.

“[In pancreatic cancer] you might want to use high-dose radiation for locally advanced disease, but the pancreas is too close to other organs that cannot handle the side effects of radiation,” Riley said. “We are currently studying GC4419’s anti-tumor effect in a Phase 1/2 clinical trial in pancreatic cancer at MD Anderson Cancer Center.”

https://bit.ly/2wmXNVN

After data, Kyowa advances Parkinson’s plan despite being dropped by Lundbeck

Kyowa Hakko Kirin has posted (PDF) a look at proof-of-concept data on adenosine A2A receptor antagonist KW-6356 in early Parkinson’s disease patients. The data emboldened Kyowa to commit to a larger phase 2 trial, despite its one-time partner Lundbeck walking away from the program.

Tokyo-based Kyowa outlicensed the ex-Asia rights to KW-6356 to Lundbeck in 2010 and went on to move the drug into a phase 2 trial in Japan in 2016. Top-line results from the 12-week, 168-patient trial are now in.

Subjects in the high- and low-dose KW-6356 arms experienced improvements on a Parkinson’s motor examination scale of of 4.76 and 5.37, respectively. The scores in the placebo arm improved by 3.14. A 2015 paper found an improvement of 3.25 is needed for clinically pertinent changes to occur, suggesting the gains seen in the treatment arms were meaningful to the lives of the patients.

Kyowa has interpreted the data positively and stepped up plans to initiate a phase 2 trial before the end of the year. However, while that study will keep the program motoring forward in Japan, the fate of the drug is less clear outside of Asia.

Lundbeck held the rights to KW-6356 in these global markets but has opted to return them to Kyowa. Having regained all rights to the drug, Kyowa has decided “to go forward independently with the review of measures to maximize value both in Japan and globally.”

Kyowa sees KW-6356 as the successor to Nouriast, another adenosine A2A receptor antagonist. The FDA rejected a filing for approval of Nouriast in 2008 amid concerns about its clinical utility. Kyowa bounced back by winning approval in Japan in 2013, only for a phase 3 flop to dial up doubts about the drug’s efficacy. Despite the phase 3 failure, Kyowa is preparing to file an NDA with the FDA.

Adenosine A2A receptor antagonists have been problematic for other companies, too. Merck took an asset deep into the clinic before data suggested it was no more effective than placebo. More recently, Acorda Therapeutics scrapped development of tozadenant after five people died of sepsis.

The travails of these and other programs means there remains a need for better Parkinson’s drugs. Kyowa thinks it has such a drug on its hands, and is forging ahead solo.

https://bit.ly/2L9YerJ

Alexion gets priority review for rare blood disorder med

Alexion Pharmaceuticals Inc. (NASDAQ:ALXN) announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the Company’s Biologics License Application (BLA) for approval of ALXN1210, the Company’s investigational long-acting C5 complement inhibitor, for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). The FDA set a Prescription Drug User Fee Act (PDUFA) date of February 18, 2019, as part of an expedited eight-month review instead of the standard 12-month review following Alexion’s use of a rare disease priority review voucher. The application is supported by comprehensive data from two rigorous Phase 3 clinical trials.

“We are working with the FDA to facilitate a smooth review,” said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. “Building on comprehensive results from the largest-ever Phase 3 development program in PNH, 11 years of proven efficacy and safety with Soliris^®, and 25 years of leadership in complement biology, we are on track with our efforts to establish ALXN1210 as the new standard of care for patients with PNH.”

If approved, ALXN1210 would be the first and only long-acting complement inhibitor for patients with PNH, providing immediate and complete inhibition of the C5 complement protein that is sustained over an eight-week dosing interval. The Phase 3 clinical development program of ALXN1210 is the largest-ever Phase 3 program in PNH. The studies enrolled a very broad and diverse population of more than 440 patients, which included patients who had never been treated with a complement inhibitor and patients who were stable on Soliris^® and switched to ALXN1210. Topline data were disclosed in press releases on March 15, 2018 and April 26, 2018, respectively.

In addition to the submission in the U.S. on June 18 and the submission in the European Union (EU) on June 28, Alexion is preparing a submission for a New Drug Application for ALXN1210 as a treatment for patients with PNH in Japan in the second half of the year. The European Medicines Agency (EMA) has accepted and is reviewing the submission for the EU. ALXN1210 has received Orphan Drug Designation (ODD) in the U.S. and EU for the treatment of patients with PNH.

Paroxysmal nocturnal hemoglobinuria (PNH) is a chronic, progressive, debilitating, and potentially life-threatening ultra-rare blood disorder that can strike men and women of all races, backgrounds, and ages without warning, with an average age of onset in the early 30s.^1,2,3 PNH often goes unrecognized, with delays in diagnosis ranging from one to more than 10 years.² In patients with PNH, chronic, uncontrolled activation of the complement system, a component of the body’s immune system, results in hemolysis (the destruction of red blood cells)⁴, which in turn can result in progressive anemia, fatigue, dark urine, and shortness of breath.^5,6,7 The most devastating consequence of chronic hemolysis is thrombosis (the formation of blood clots), which can damage vital organs and cause premature death.⁸ Historically, it had been estimated that one in three patients with PNH did not survive more than five years from the time of diagnosis.² PNH is more common among patients with disorders of the bone marrow, including aplastic anemia (AA) and myelodysplastic syndromes (MDS).^9,10,11 In certain patients with thrombosis of unknown origin, PNH may be an underlying cause.⁴

https://bit.ly/2PpZ8Um

Consortium of investors proposes to acquire China Biologic for $118 per share

A consortium composed of David Gao, GL Capital Group, Bank of China Group Investment Limited, and CDH Investments announced it has made a non-binding proposal to acquire all of the outstanding common shares of China Biologic Products Holdings, that are not already held by the Consortium, for $118.00 per share in an all-cash transaction valued at approximately $3.9B. The proposal was conveyed on August 17, 2018 in a letter to China Biologic’s board. The Consortium’s proposal represents an approximate 30% premium to China Biologic’s closing share price on August 16, 2018, and an approximate 40% premium to the company’s closing price on June 8, 2018, the last trading day before CITIC Capital’s proposal to acquire the company for $110.00 per share was conveyed.

https://bit.ly/2nQZLd5

Tandem Diabetes price target raised to $60 from $35 at Lake Street

Lake Street analyst Brooks O’Neil affirmed a Buy rating on Tandem Diabetes Care (TNDM) and raised his price target to $60 from $35 following the U.S. commercial launch of its t:slim X2 insulin pump with Basal-IQ, which is integrated with Dexcom’s (DXCM) G6 CGM. In a research note to investors, O’Neil contends that while the stock is up 36.4% in the 14 trading days since its Q2 earnings release, he believes it is “just getting going.”

https://bit.ly/2vWHIXm

Aslan Pharmaceuticals gets orphan drug tag for leukemia med

ASLAN Pharmaceuticals announced that the FDA has granted ASLAN003 Orphan Drug Designation, or ODD, as a treatment for acute myeloid leukaemia, or AML. ASLAN003 is an orally active, potent inhibitor of human dihydroorotate dehydrogenase that has the potential to be first-in-class in AML. AML is a cancer of the myeloid line of blood cells, characterised primarily by the rapid growth of abnormal white blood cells that build up in the bone marrow and interfere with the production of normal blood cells.

https://bit.ly/2L4yUD